Ian Phillips Publishes Review of Gene and Stem Cell Therapies

September 25, 2012

In a recently published paper in the Nature journal, Clinical and Pharmacological Therapeutics, Dr. Ian Phillips analyzed the recent progress of stem cell and gene therapy for rare diseases. In the paper, Phillips, who is the Norris Professor of Applied Science and director of the Center for Rare Disease Therapies (CRDT) at KGI, summarizes ongoing gene therapy trials in humans with rare diseases, including, macular degeneration, metastatic melanoma, Duchenne muscular dystrophy, and Leber's congenital amaurosis (LCA), which is a rare inherited eye disease that can cause severe vision loss or blindness. Of these, one of the most dramatic is LAC blindness which can be treated by a single injection of a normal gene to replace a mutated gene in the retina. He also examines stem cell therapy for many of the same rare diseases and describes future methods of taking cells from the skin of a patient with a disease and turning their cells into stem cells. These cells can then be repaired in a dish and injected back into the patient to treat the disease. Human stem cells, he noted, can also be used for testing new drugs and locating the source of the genetic problem.

“All this progress indicates a bright future for gene and stem cell therapies in medicine, especially where there are no current therapies or drugs available,” Phillips said. “However, a cure with a single injection of genes or stem cells will be a challenge to companies deciding on a price for commercializing these therapies.”

Phillips also recently spoke at Pfizer's first Gene Therapy Workshop held at the company's Specialty Care Business Unit campus in Collegeville, Pennsylvania, on September 12. During the talk, he traced the history and theory of viral delivery for gene therapy and described the progress being made towards developing viable gene therapies. He also led a discussion on new developments in gene therapy for rare diseases, including hemophilia B and a rare lipid dystrophy disease, among others. The workshop was organized by Dr. May Orfali, senior director of the Rare Disease Unit at Pfizer. Dr. Orfali was the liaison for last year's Pfizer-sponsored Team Master Project which compared key elements of drug development in orphan vs. non-orphan drugs in specialty medicine.